Date: Wed, 5 Apr 95 14:54:13 -0400 From: Bob Broedel To: als@huey.met.fsu.edu Subject: ALSD183 ALS-ON-LINE =============================================================== == == == ----------- ALS Interest Group ----------- == == ALS Digest (#183, 05 April 1995) == == == == ------ Amyotrophic Lateral Sclerosis (ALS) == == ------ Motor Neurone Disease (MND) == == ------ Lou Gehrig's disease == == ------ Charcot's Disease == == == == This e-mail list has been set up to serve the world-wide == == ALS community. That is, ALS patients, ALS researchers, == == ALS support/discussion groups, ALS clinics, etc. Others == == are welcome (and invited) to join. The ALS Digest is == == published (approximately) weekly. Currently there are == == 610+ subscribers. == == == == To subscribe, to unsubscribe, to contribute notes, == == etc. to ALS Digest, please send e-mail to: == == bro@huey.met.fsu.edu (Bob Broedel) == == Sorry, but this is *not* a LISTSERV setup. == == == == Bob Broedel; P.O. Box 20049; Tallahassee, FL 32316 USA == =============================================================== CONTENTS OF THIS ISSUE: 1 .. Riluzole/Rilutek 2 .. Riluzole Study Results Are In! 3 .. Riluzole & ALS 4 .. Rhone-Poulenc ALS Drug Has Modest Effect 5 .. Cephalon/Myotrophin 6 .. Myotrophin & ALS 7 .. re: swallowing problems 8 .. CytoTherapeutics 9 .. primary lateral sclerosis 10 . Auction to Benefit ALS Support 11 . ALS Fund Raising (1) ===== Riluzole/Rilutek ========== Date : Tue, 4 Apr 1995 20:11:50 -0500 (CDT) >From : HEINET@wartburg.edu Subject: Riluzole/Rilutek Prior to its 9amEDT press release on the results of the clinical trial of Rilutek/Rioluzole, Rhone-Poulenc Rorer held a telephone conference call with the five ALS patients who had participated in the Feb 13 focus group meeting. Patients are: Susan Pinson, Mark Malconian, Mariah Gladdis, Jack Norton, and Ted Heine. Purpose of the conference call was to alert the patients to the contents of the forthcoming press release, so we received no news beyond the press release contents. However, it was a great gesture of goodwill on the part of RPR, and an indication of its intent to focus on patient welfare. All of us extended our thanks to RPR on behalf of the worldwide community of ALS patients. We have the knowledge that this company, from the CEO on down, really cares and is doing its very best to expedite drug availability, both in compassionate access protocols as well as in ultimate marketing of an approved drug. Ted Heine in Waverly, Iowa (2) ===== Riluzole Study Results Are In! ========== Date : Tue, 4 Apr 1995 11:19:38 -0500 >From : jacobson@phoenix.phoenix.net (Doug Jacobson) Subject: Riluzole Study Results Are In! Bro, and everyone else, I have some VERY good news. I just got off the phone with Dr. Harati here in Houston, and he said that the riluzole trial results were discussed in Paris, and they are as follow: The trials that were examined, a smaller European trial, and the large trial in the US, both showed that riluzole increased survival in patients by about 12%. It was effective on both limb- and bulbar-onset patients. The drug did not increase muscular function or strength. RPR is working with the FDA to set up an early compassionate use release of the drug as early as July 1995 (although this is subject to change). I'm sure that this will hit the media today or tomorrow. Doug (3) ===== Riluzole & ALS ========== Tuesday April 04, 1995 IMPACT OF RILUTEK (TM) (RILUZOLE) ON SURVIVAL IN AMYOTROPHIC LATERAL SCLEROSIS (ALS) CONFIRMED IN MULTINATIONAL PHASE III TRIAL COLLEGEVILLE, Pa., and Paris /PRNewswire/ -- Rhone-Poulenc Rorer Inc. (NYSE: RPR) announced today the results of its preliminary analysis of a Phase III clinical trial involving over 950 people with Amyotrophic Lateral Sclerosis (ALS). These results generally confirm the findings of a previous Phase II clinical trial demonstrating that Rilutek is the first medicine to show some impact on survival for ALS patients. These Phase III results will be presented at the American Academy of Neurology meeting in early May. The previous Phase II clinical trial that was published in the March 3, 1994, issue of the New England Journal of Medicine concluded that ALS patients who received Rilutek experienced an overall average increase in survival of approximately three months. While further statistical evaluation of the Phase III trial is ongoing, an effect on survival was observed in the overall treated patient population, which included both bulbar and limb onset ALS patients. The initial analysis did not show a statistically significant effect on the rate of muscle function deterioration for the overall ALS population. The Phase III clinical trial was a multinational double-blind, placeo-controlled study conducted at 31 investigator sites in Europe and North America. This study, involving over 950 patients, the largest ever in ALS, was conducted in collaboration with a Steering Committee and an independent Data Safety Monitoring Board. Enrollment began in December 1992 and the study was completed in December 1994. The participating centers in the United States were Baylor College of Medicine and Veterans Affairs Medical Center (Houston), Johns Hopkins University (Baltimore), Northwestern University (Chicago), California Pacific Medical Center (San Francisco) and Tufts-New England Medical Center (Boston). Efficacy and safety parameters were compared among four groups: three doses of Rilutek in the amount of 50 mg, 100 mg, 200 mg (total daily dose) or placebo. A total of 75 percent of the patients received Rilutek. The primary endpoint of the study was survival. The treatment duration was up to 18 months, after which all patients were given the opportunity to receive Rilutek 100 mg/day on an open-label basis. Side effects reported in the trial included nausea, asthenia (fatigue), and elevated liver enzymes. "The results represent an important first step in the treatment of ALS. Our top priority now is to work closely with regulatory authorities to make Rilutek available to ALS patients worldwide as soon as possible by applying for regulatory approvals," said Manfred Karobath, M.D., Senior Vice President and President of Research and Development at RPR. "RPR will utilize all necessary resources to submit registration dossiers to regulatory authorities, beginning in July 1995." Since no other treatment has demonstrated efficacy in, or has been approved for ALS, RPR has begun discussions with local regulatory authorities to initiate locally approved programs that would allow broader access to Rilutek during the course of regulatory reviews. In the United States, RPR is planning to submit a Treatment IND (investigational new drug) Application this month to the Food and Drug Administration. Worldwide, the annual incidence of ALS is estimated to be 2.5 new cases per 100,000 people. ALS is also known as Lou Gehrig's disease, maladie de Charcot, or Motor Neurone Disease. Generally, patients survive three to five years after diagnosis. Death usually occurs due to respiratory failure. The outward signs of ALS are progressive weakness and deterioration of the muscles (amyotrophy), beginning in the limbs, usually on one side of the body (lateral). Inside the body, the nerves controlling motor function die off after their cell bodies become hardened and shriveled (sclerosis), leaving the patient increasingly helpless. Limb onset ALS occurs when there is damage to motor neurons in the spinal cord that run to limb muscles. The muscles used for everyday activities are affected first. In bulbar onset ALS, damage occurs to the motor neurons of the bulbar region of the brain, which weakens the muscles responsible for vital bodily functions, such as breathing, swallowing, and talking. For more information, patients, caregivers or health care providers can call 1-800 RX TRIAL (1-800-798-7425). Rhone-Poulenc Rorer is a global pharmaceutical company dedicated to improving human health. CONTACT: Bob Pearson, 610-454-3872, or Guy Esnouf, 610-454-5048, both of Rhone-Poulenc Rorer (4) ===== Rhone-Poulenc ALS Drug Has Modest Effect ========== WALL STREET JOURNAL 04/05/95 Technology & Health: Rhone-Poulenc ALS Drug Has Modest Effect By Michael Waldholz, Staff Reporter of The Wall Street Journal Rhone-Poulenc Rorer Inc. said a test of its experimental drug provided a modest increase in survival for patients with the degenerative nerve disorder amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig's disease. < parts deleted > "That there may now be something, although small and modest, is a significant step forward," said Lynn Klein, vice president for patient services of the Amyotrophic Lateral Sclerosis Association, Woodland Hills, Calif. "We consider this a very, very exciting development." Several doctors who took part in the drug trial urged caution in evaluating the results. "Patients taking this drug should not expect a recovery or even a significant change in the course of their disease," said Yadollah Harati, a professor of neurology at Baylor College of Medicine and the Veterans Affairs Medical Center in Houston. Dr. Harati said none of the patients in his trial appeared to have a significant improvement. "I wasn't able to tell who was taking the drug," he said. < parts deleted > Company officials and study doctors said they hoped Rilutek might someday be part of a therapy of several useful drugs. Cephalon Inc., a biotechnology company in West Chester, Pa., said on Monday that it's evaluating the results of a large trial of its drug, Myotrophin, which works in a different way against the disease. Cephalon's test results will be released in June. < rest of article deleted > (5) ===== Cephalon/Myotrophin ========== Date : Tue, 4 Apr 1995 20:16:32 -0500 (CDT) >From : HEINET@wartburg.edu Subject: Cephalon/Myotrophin On April 3 (obviously to beat Rhone-PoulencRorer's April 4 announcement) Cephalon sent out a press release to the effect that it had completed PhaseII/III clinical trials of Myotrophin (Insulin-like growth factor 1) and that it will announce results at a neuro conference in Paris in June. I think this implies positive results, or it would have acted as did Regeneron and Amgen/Synergen in announcing disappointing results publicly and cancelling its trial. Ted Heine in Waverly, Iowa (6) ===== Myotrophin & ALS ========== CEPHALON COMPLETES INITIAL PHASE II/III CLINICAL TRIAL OF MYOTROPHIN(TM) IN ALS WEST CHESTER, Pa., April 3 /PRNewswire/ -- Cephalon, Inc. (Nasdaq: CEPH) announced today the completion of its North American Phase II/III clinical trial of Myotrophin(TM) (rhIGF-1) for the treatment of amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease). This double-blind, placebo- controlled study of 266 patients with ALS was conducted at eight medical centers in the United States and Canada to evaluate the drug's potential to treat the disease. The company has begun the process of unblinding all safety and efficacy information obtained during the study and expects to begin data analysis shortly. Cephalon plans to present the study results at the World Federation of Neurology Workshop on Therapeutic Trials in ALS in Talloires, France, June 8-10, 1995. Cephalon is also conducting a similar Phase II/III clinical study of Myotrophin in 183 patients with ALS in Europe. That study is expected to conclude this summer, with results to be made available after completion of data analysis. < parts deleted > Cephalon is developing Myotrophin in the United States and Europe for use in ALS in collaboration with Chiron Corporation (NASDAQ: CHIR), and for Cephalon Clinical Partners, L.P., which was formed in 1992 to fund development and clinical testing of the drug. Cephalon is developing Myotrophin in Japan for use in ALS and other neurological disorders in collaboration with Kyowa Hakko Kogyo Co., Ltd. < parts deleted > CONTACT: Jason Rubin or Mary Fisher, of Cephalon, 610-344-0200 (7) ===== re: swallowing problems ========== Date : Sat, 25 Mar 1995 01:01:15 -0500 >From : DanB5974@aol.com Subject: re: Swallowing problems I'm just passing this along. I have been sharing the ALS digest with my friend Robert, who has ALS. When I got to the part about swallowing difficulties, he got on the soapbox about Valium. He's been taking Valium for a long time now, and feels it extended his oral abilities so much he beleives it is well worth while. (He still takes it, but can no longer eat. He does somewhat talk.) Considering his history, it's plausable that it has extended and preserved his oral abilities. But it's hard to say for sure. I think what he's saying is that he was rapidly losing his voice a couple years ago, then the degression came to a screaching halt when he started using Valium. Comments from researchers/experiences? (8) ===== CytoTherapeutics ========== Date : Tue, 4 Apr 1995 20:22:50 -0500 (CDT) >From : HEINET@wartburg.edu Subject: CytoTherapeutics CytoTherapeutics is the company that announced ten days ago that it planned to implant tiny membrans that contained genes producing CNTF in the spinal columns of ten ALS patients in Switzerland. The April 3 Wall Street Journal had an article on the company that talked about its technology but did not mention ALS. As a layperson, this procedure to introduce CNTF directly into the spinal fluid strikes me as less expensive and less risky than the intrathecal pump procedure being tried on a few patients in the US. However, will either one work???? Ted Heine in Waverly, Iowa (9) ===== primary lateral sclerosis ========== Date : Tue, 28 Mar 1995 11:05:07 -0600 (CST) >From : marjorie sablik x339 Subject: primary lateral sclerosis In a recent PLS newsletter, there was a write-up about a drug called C-3036 for the treatment of primary lateral sclerosis. Does anybody have any information regarding what this drug is and where it can be obtained? Is there any literature references to this? Please send any information to msablik@icarus.sfbr.org Thank you, Marjorie (10) ===== Auction to Benefit ALS Support ========== PALACE OF AUBURN HILLS TO HOST SPORTS MEMORABILIA AUCTION TO BENEFIT A.L.S. OF MICHIGAN, INC. DETROIT, March 29 /PRNewswire/ -- The 2nd Annual A.L.S. of Michigan Sports Memorabilia Auction will take place on April 12, 1995, immediately following the 7:30 p.m. Pistons vs. Chicago Bulls game at the Palace of Auburn Hills, MI. Pistons play-by-play announcer George Blaha will act as Auctioneer. Last year over 600 spectators stayed after a Piston's game to attend the auction. This year, with Michael Jordan's return to the Bulls, attendance at the auction is expected to double. < parts deleted > All proceeds will go toward maintaining the patient services programs of A.L.S. of Michigan. < parts deleted > CONTACT: Jennifer Badnarik, Communications Manager of A.L.S. of Michigan, 313-933-3257 (11) ===== ALS fund raising ========== NO FOOLIN' -- LAWN MOWER RACING SEASON BEGINS APRIL 1! ROCKFORD, ILL., WILL HOST 4TH ANNUAL STA-BIL NATIONALS GLENVIEW, Ill., March 29 /PRNewswire/ -- April Fool's Day traditionally marks the opening of lawn mower racing season and thus announcement by the U.S. Lawn Mower Racing Association (USLMRA) that the 4th annual STA-BIL Nationals will take place on Saturday, Sept. 2, at the Rockford MetroCentre in Rockford, Ill. < parts deleted > Race entry fees from The STA-BIL Nationals benefit the Les Turner ALS Foundation, the nation's largest independent organization dedicated to finding a cause and cure for amyotrophic lateral sclerosis, or Lou Gehrig's disease. The Nationals have attracted 5,000 lawn mower racing fans and raised $12,000 for ALS since inception in 1992. < parts deleted > CONTACT: Bruce Kaufman or Dave Silbar of Merton G. Silbar Public Relations, 708-729-7363, for U.S. Lawn Mower Racing Association === end of als 183 ===